In a groundbreaking study, researchers found lithium—a medicine normally used to treat bipolar disorder—as a viable therapy for autism spectrum disorder (ASD). The study, conducted by the Institute for Basic Science (IBS) in South Korea, found that lithium could treat some of the basic symptoms of ASD by restoring brain function and alleviating behavioural symptoms in mice with Dyrk1A gene mutations, a known risk factor for the illness. 

ASD is a neurodevelopmental disorder that affects about 2.8% of the world's population. It is characterised by social difficulties, repetitive behaviours, intellectual issues, and anxiety. Despite its broad prevalence, there are no definitive therapies for autism spectrum disorders. The discovery that lithium may have therapeutic benefits opens up new possibilities for targeting the disorder's basic mechanisms.

The study, which was led by IBS Director Kim Eunjoon and the Centre for Synaptic Brain Dysfunctions, gave lithium to young mice with Dyrk1a mutations at a very important time in their brain development. The findings were startling: lithium not only normalised brain size but also increased neural connections, which greatly reduced feelings of anxiety and social interaction. 

The Dyrk1a gene mutation, associated with autism spectrum disorder, is the focal point of the finding. Specifically, the mutation affects brain connections, resulting in developmental delays and social deficits. Researchers revealed that the mutation alters the phosphorylation of mTOR (mammalian target of rapamycin), a vital signalling system in the brain. This disruption leads to the developmental and behavioural difficulties that come with ASD. 

The researchers looked to lithium, traditionally used to stabilise mood in bipolar patients, to explore potential treatments. The researchers selected lithium due to its ability to impact various signalling pathways, one of which is mTOR. The study discovered that giving lithium to juvenile mice with the Dyrk1a mutation restored the phosphorylation of proteins involved in the mTOR pathway, which helped to restore normal brain function. 

Furthermore, researchers discovered that lithium's influence on Kalirin-7, a protein necessary for synapse structure and function, mediates its therapeutic benefits. Kalirin-7 is essential for keeping the brain's signalling networks balanced. By focussing on this molecule, lithium helped to reconnect brain cells, which was "clearing the roadblocks" caused by the Dyrk1a mutation. 

Dr. Roh Junyeop, a senior researcher and co-first author of the study, compared the effects of the Dyrk1a mutation to a traffic jam in the brain: "Dyrk1a mutations disrupt neuronal connections, much like traffic jams or barriers in a city. Lithium helps to relieve the congestion, allowing neurones to communicate more smoothly." 

The long-term influence of lithium is one of the study's most hopeful findings. Only during the juvenile period did the short-term medication's effects persist throughout adulthood. This shows that early intervention with lithium may have long-term benefits, allowing for structural and functional brain healing and potentially lowering the severity of ASD symptoms over time. 

"These findings underscore the importance of early intervention during critical developmental windows," stated Director Kim Eunjoon. Our findings imply that we could potentially use lithium, a widely used medicine for bipolar disorder, to treat ASD. The fact that its effects last long after treatment is especially fascinating. 

The findings of this study, published in Molecular Psychiatry on December 5, provide promise for novel therapy options for ASD. Lithium's capacity to address basic ASD symptoms, such as social impairments, anxiety, and repetitive behaviours, may provide respite to many families and individuals suffering from the illness. It also emphasises the importance of early detection and intervention in controlling ASD. 

Now that this study is over, more research can be done on lithium's therapeutic potential for ASD. This could lead to specific medicines that target the molecular causes of the disorder. The findings highlight how, with further research, existing medications such as lithium could be repurposed to treat neurodevelopmental problems, providing new hope for patients who presently have few treatment options. 

Treating ASD with lithium is a promising advancement in the field of neurodevelopmental disorders, offering the potential to alleviate some of the most challenging symptoms of ASD and paving the way for future enhanced therapies.